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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Recruiting Phase 1 Interventional

CD4^LVFOXP3 in Participants With IPEX

NCT05241444
View directory record Official record
Conditions
IPEX
Interventions
CD4^LVFOXP3
Biological
Lead sponsor
Bacchetta, Rosa, MD
Other
Eligibility
4 Months to 35 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2037
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated May 21, 2025 · Synced Sep 9, 2026, 7:24 AM EDT
Terminated Phase 2Phase 3 Interventional

Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis

NCT00073463
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
tgAAVCF
Genetic
Lead sponsor
Targeted Genetics Corporation
Industry
Eligibility
12 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
11
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2008 · Synced Sep 9, 2026, 7:24 AM EDT
Completed No phase listed Observational

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

NCT05044845
View directory record Official record
Conditions
Gene Therapy, Hemophilia B
Interventions
Interview
Behavioral
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
12 Years and older
Enrollment
150 participants
Timeline
2022 – 2025
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting Phase 1Phase 2 Interventional

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316
View directory record Official record
Conditions
Fabry Disease
Interventions
AMT-191
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 50 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
8
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Sep 9, 2026, 7:24 AM EDT
Terminated Phase 1 Interventional

Gene-Modified T Cells, Vaccine Therapy, and Ipilimumab in Treating Patients With Locally Advanced or Metastatic Malignancies

NCT02070406
View directory record Official record
Conditions
Unspecified Adult Solid Tumor, Protocol Specific
Interventions
cyclophosphamide, fludarabine phosphate, NY-ESO-1 reactive TCR retroviral vector transduced autologous PBL, dendritic cell vaccine therapy, aldesleukin, fludeoxyglucose F 18, positron emission tomography, laboratory biomarker analysis
Drug · Biological · Radiation + 2 more
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
16 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 27, 2019 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Therapy for Achromatopsia (CNGA3)

NCT03758404
View directory record Official record
Conditions
Achromatopsia
Interventions
adeno-associated virus vector AAV- CNGA3
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 30, 2022 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 1 Interventional

Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

NCT01161576
View directory record Official record
Conditions
Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies, AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 18 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2020
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 1, 2021 · Synced Sep 9, 2026, 7:24 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration

NCT05984927
View directory record Official record
Conditions
Age-Related Macular Degeneration
Interventions
NG101 AAV gene therapy
Genetic
Lead sponsor
Elisigen, Inc.
Industry
Eligibility
50 Years to 89 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
2
States / cities
Cincinnati, Ohio • Bellaire, Texas
Source: ClinicalTrials.gov public record
Updated Jan 19, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 1 Interventional

AAVCAGsCD59 for the Treatment of Wet AMD

NCT03585556
View directory record Official record
Conditions
Wet Age-related Macular Degeneration
Interventions
Intravitreal anti-VEGF, Intravitreal AAVCAGsCD59, Oral prednisolone
Drug · Biological
Lead sponsor
Janssen Research & Development, LLC
Industry
Eligibility
50 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Boston, Massachusetts • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 1 Interventional Results available

T Cells Expressing a Fully-human AntiCD19 Chimeric Antigen Receptor for Treating B-cell Malignancies

NCT02659943
View directory record Official record
Conditions
Lymphoma, B-Cell, Lymphoma, Non-hodgkins
Interventions
Anti-cluster of differentiation 19 (CD19)-Chimeric Antigen Receptor (CAR) T cells, Cyclophosphamide, Fludarabine
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 73 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 18, 2023 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 1 Interventional

Safety Study of AADC Gene Therapy (VY-AADC01) for Parkinson's Disease

NCT01973543
View directory record Official record
Conditions
Parkinson's Disease
Interventions
VY-AADC01
Biological
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
40 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
2
States / cities
San Francisco, California • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2020 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 3 Interventional Results available

RESCUE and REVERSE Long-term Follow-up

NCT03406104
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Sham Intravitreal Injection
Genetic · Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Pasadena, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

NCT01687608
View directory record Official record
Conditions
Hemophilia B
Interventions
AskBio009
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
15
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

NCT07135050
View directory record Official record
Conditions
Pitt Hopkins Syndrome
Interventions
MZ-1866
Genetic
Lead sponsor
Mahzi Therapeutics
Industry
Eligibility
2 Years to 25 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
3
States / cities
Oakland, California • Aurora, Colorado • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Terminated No phase listed Observational

Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer

NCT00533572
View directory record Official record
Conditions
Epidermolysis Bullosa Dystrophica
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 26, 2015 · Synced Sep 9, 2026, 7:24 AM EDT
Completed No phase listed Observational

Genes Involved in Lipid Disorders

NCT02311335
View directory record Official record
Conditions
Lipid Disorders
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 2 Interventional

RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)

NCT04832724
View directory record Official record
Conditions
Neovascular Age-related Macular Degeneration, Wet Macular Degeneration, Wet Age-related Macular Degeneration
Interventions
RGX-314
Genetic
Lead sponsor
AbbVie
Industry
Eligibility
50 Years to 89 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
13
States / cities
Phoenix, Arizona • Sun City, Arizona • San Diego, California + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2024 · Synced Sep 9, 2026, 7:24 AM EDT
Recruiting Phase 1Phase 2 Interventional

Gene Therapy for CD19-Positive Hematologic Malignancies (SENTRY-CD19)

NCT06533579
View directory record Official record
Conditions
B-cell Acute Lymphoblastic Leukemia, Large B-cell Lymphoma, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone Lymphoma, Follicular Lymphoma, Mantle Cell Lymphoma, Diffuse Large B Cell Lymphoma, High-grade B-cell Lymphoma, Burkitt Lymphoma, Primary Mediastinal Large B-cell Lymphoma (PMBCL), Non Hodgkin Lymphoma, Mixed Phenotype Acute Leukemia
Interventions
Dose Level 1, VNX-101, Dose Level 2, VNX-101, Dose Level 3, VNX-101, Dose Level 4, VNX-101
Genetic
Lead sponsor
Vironexis Biotherapeutics Inc.
Industry
Eligibility
13 Years to 90 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
11
States / cities
Duarte, California • Los Angeles, California • Denver, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Withdrawn Phase 1Phase 2 Interventional

A Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA

NCT05778877
View directory record Official record
Conditions
Methylmalonic Acidemia (MMA)
Interventions
SEL-302
Drug
Lead sponsor
Selecta Biosciences, Inc.
Industry
Eligibility
3 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency

NCT02168686
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
ADVM-043
Genetic
Lead sponsor
Adverum Biotechnologies, Inc.
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Gainesville, Florida • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Sep 9, 2026, 7:24 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

NCT02906202
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 17, 2023 · Synced Sep 9, 2026, 7:24 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia

NCT05926765
View directory record Official record
Conditions
Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands
Interventions
AAV2-hAQP1 Concentration 1, AAV2-hAQP1 Concentration 2, Placebo, AAV2-hAQP1 Concentration 3, AAV2-hAQP1 Concentration 4
Genetic · Other
Lead sponsor
MeiraGTx, LLC
Industry
Eligibility
18 Years and older
Enrollment
290 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
18
States / cities
Gilbert, Arizona • Duarte, California • Miami, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Sep 9, 2026, 7:24 AM EDT
Completed No phase listed Observational

Bone Marrow for Hemoglobinopathy Research

NCT00669305
View directory record Official record
Conditions
Sickle Cell Anemia, Thalassemia
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 24, 2020 · Synced Sep 9, 2026, 7:24 AM EDT
Terminated Phase 1 Interventional

Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

NCT06747273
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Interventions
SRP-9004
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
2
States / cities
Columbus, Ohio • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced Sep 9, 2026, 7:24 AM EDT